Introduction
In 2014, the European Medicines Agency (EMA), granted a ‘Conditional Marketing Authorisation’ to Translarna for the treatment of patients with Duchenne muscular dystrophy caused by a nonsense mutation who are aged two years and older and ambulatory (able to walk).
The UK was still in the European Union (EU) and part of the EMA regulatory system at that time, and therefore Translarna has been available to eligible patients in the UK since then. It was available through a Managed Access Agreement until January 2023, when a full NICE appraisal recommended Translarna be made available through the NHS as an effective and cost-effective treatment. You can read more about the NICE guidance on the NICE website.
Post-Brexit, in England, Wales and Scotland decisions on marketing authorisation are the responsibility of the UK Medicines and Healthcare products Regulatory Agency (MHRA).
The company that makes the treatment, PTC Therapeutics, has approached the MHRA asking for an independent evaluation of Translarna to renew the conditional marketing authorisation in the UK.
As part of the assessment process the MHRA is seeking input from patient organisations, and Muscular Dystrophy UK and Action Duchenne are working together to gather the views of individuals and families with experience of receiving Translarna.
This survey is about your experience of receiving Translarna; or the experience of someone you care for. Completing the survey will help us to represent the views of patients and families to MHRA, who are meeting at the end of June to discuss the treatment.
Please note that the MHRA process is entirely separate from the EMA process. In September 2023 the EMA’s Committee for Medicinal Products for Human Use (CHMP) released a recommendation not to renew the approval (authorisation) for the marketing of Translarna. On 20 May 2024 PTC Therapeutics announced that the European Commission (EC) has decided not to adopt this recommendation and has asked CHMP for a re-evaluation. You can read more about this in PTC’s press release. The MHRA review of Translarna is continuing.
If you or someone you care for has experience of receiving Translarna, please complete this survey by midnight on 2 June.
